220 patents
Page 5 of 11
Utility
Compounds and Methods for Use in Dystrophin Transcript
17 Mar 22
Provided herein are methods, compounds, and compositions for modulation of dystrophin pre-mRNA in an animal.
Frank Rigo, Thazha P. Prakash, Punit P. Seth
Filed: 18 Nov 20
Utility
Methods and Compositions for Modulating ALPHA-1-ANTITRYPSIN Expression
10 Mar 22
Disclosed herein are methods for decreasing A1AT mRNA and protein expression and treating, ameliorating, preventing, slowing progression, or stopping progression of fibrosis.
Brett P. Monia, Michael L. McCaleb, Susan M. Freier, Shuling Guo
Filed: 13 Oct 20
Utility
Compounds and Methods for Modulation of SMN2
10 Mar 22
Disclosed herein are compounds, compositions and methods for modulating splicing of SMN2.
Frank Rigo, Thazha P. Prakash, Punit P. Seth
Filed: 18 Dec 20
Utility
Compositions and Methods for Treating and Preventing Amyotrophic Lateral Sclerosis
10 Mar 22
Dosage regimens for SOD1-targeting antisense oligonucleotides, and salts thereof, are provided.
Ivan Alexandrov Nestorov, Toby Ferguson, Daniel A. Norris
Filed: 12 Dec 19
Utility
Modified Oligomeric Compounds and Uses Thereof
3 Mar 22
The present disclosure provides oligomeric compounds comprising a modified oligonucleotide having at least one stereo-non-standard nucleoside.
Punit P. Seth, Michael T. Migawa, Graeme C. Freestone
Filed: 4 Oct 19
Utility
Compounds and Methods for Reducing ATXN3 Expression
3 Mar 22
Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of ATXN3 RNA in a cell or animal, and in certain embodiments reducing the amount of ATXN3 protein in a cell or animal.
Susan M. Freier
Filed: 9 May 19
Utility
Compounds and Methods for Modulating SMN2
3 Mar 22
Provided are compounds, methods, and pharmaceutical compositions for modulating SMN2 RNA and/or protein in a cell or subject.
Frank Rigo, Thazha P. Prakash, Kar Yun Karen Ling, W. Brad Wan, William John Drury, III
Filed: 26 Feb 21
Utility
Compounds and Methods for Reducing ATXN2 Expression
3 Mar 22
Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of ATXN2 RNA in a cell or animal, and in certain instances reducing the amount of Ataxin-2 protein in a cell or animal Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a neurodegenerative disease.
Susan M. Freier, Priyam Singh, Frank Rigo, Paymaan Jafar-nejad, Holly Kordasiewicz
Filed: 23 Apr 21
Utility
Modulation of Hepatitis B Virus (HBV) Expression
3 Mar 22
Disclosed herein are antisense compounds and methods for decreasing HBV mRNA, DNA and protein expression.
Eric E. SWAYZE, Susan M. Freier, Michael L. McCaleb
Filed: 30 Jul 21
Utility
Compounds and Methods for Modulating PLP1
24 Feb 22
Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of PLP1 RNA in a cell or subject, and in certain instances reducing the amount of proteolipid protein 1 in a cell or subject.
Berit Elissa Powers
Filed: 29 Jun 21
Utility
Modulators of IRF5 Expression
17 Feb 22
The present embodiments provide methods, compounds, and compositions useful for inhibiting IRF5 expression, which may be useful for treating, preventing, or ameliorating a disease associated with IRF5.
Susan M. FREIER
Filed: 3 Nov 21
Utility
Compositions and Their Uses Directed to Huntingtin
10 Feb 22
Disclosed herein are compounds, compositions and methods for modulating the expression of huntingtin in a cell, tissue or animal.
Susan M. Freier
Filed: 21 Jun 21
Utility
Compositions and Methods for Modulation of Lmna Expression
3 Feb 22
The present disclosure provides compounds comprising oligonucleotides complementary to a portion of the LMNA gene.
Priyam Singh, Frank Rigo, Tom Misteli, Madaiah Puttaraju
Filed: 20 Sep 19
Utility
Conjugated Antisense Compounds and Their Use
27 Jan 22
The present disclosure provides duplexes comprising a first oligomeric compound and a second oligomeric compound wherein the second oligomeric compound comprises a conjugate group.
Punit P. Seth, Michael Oestergaard, Eric E. Swayze
Filed: 10 Aug 21
Utility
Compounds and Methods for Reducing Prion Expression
27 Jan 22
Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of PRNP RNA in a cell or animal, and in certain instances reducing the amount of PrP protein in a cell or animal.
Susan M. Freier, Huynh-Hoa Bui, Hien Thuy Zhao
Filed: 21 Nov 19
Utility
Oligomer-conjugate Complexes and Their Use
27 Jan 22
Oligonucleotides, chemically-modified oligonucleotides, and oligonucleotide-conjugate complexes for use in research, diagnostics, and/or therapeutics are described herein.
Thazha P. Prakash, Eric E. Swayze, Walter F. Lima, Garth A. Kinberger
Filed: 1 Jul 21
Utility
Compounds and Methods for Modulating Angiotensinogen Expression
6 Jan 22
Disclosed herein are compositions and compounds comprising modified oligonucleotides for modulating AGT and modulating a RAAS pathway related disease, disorder and/or condition in an individual in need thereof.
Adam Mullick, Mark J. Graham, Punit P. Seth, Susan M. Freier
Filed: 23 Nov 20
Utility
Compounds and Methods for Modulation of Dystrophia Myotonica-protein Kinase (DMPK) Expression
30 Dec 21
Provided herein are methods, compounds, and compositions for reducing expression of a DMPK mRNA and protein in an animal.
Eric E. Swayze, Susan M. Freier
Filed: 10 Feb 21
Utility
Compositions and Methods for Modulating Apolipoprotein C-iii Expression
23 Dec 21
Provided herein are oligomeric compounds with conjugate groups targeting apolipoprotein C-III (ApoCIII).
Thazha P. Prakash, Punit P. Seth, Eric E. Swayze, Mark J. Graham
Filed: 7 Dec 20
Utility
Chirally Enriched Oligomeric Compounds
9 Dec 21
The present disclosure provides oligomeric compounds comprising a modified oligonucleotide having one or more chirally enriched phosphorothioate intemucleoside linkages.
Punit P. Seth, Michael Oestergaard, Michael T. Migawa, Xue-hai Liang, Wen Shen, Stanley T. Crooke, Eric E. Swayze
Filed: 4 Oct 19